A Single-Arm, Open-Label, Multicenter, Phase 1b/2 Study Evaluating the Safety and Efficacy of AUTO1 (Obecabtagene Autoleucel [Obe-cel]) in Pediatric Patients With CD19-positive Relapsed/Refractory (R/R) B Cell Acute Lymphoblastic Leukemia (B ALL) or R/R Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL).
Autolus Limited
Summary
This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL).
Description
This is a single-arm, open-label, multi-center, Phase 1b/2 study to determine the safety and efficacy of obe-cel administered intravenously in pediatric patients \< 18 years old with r/r B ALL and with r/r aggressive mature B NHL. The safety and tolerability of obe cel in pediatric patients will be continually monitored by the Sponsor. Efficacy endpoints will be determined by an Independent Response Review Committee (IRRC). The study will involve consented patients going through the following sequential study periods: screening, leukapheresis, bridging as necessary, lymphodepletion, treatmen…
Eligibility
- Age range
- 0–18 years
- Sex
- All
- Healthy volunteers
- No
INCLUSION CRITERIA: * \< 18 years old at screening * ≥ 6 kg body weight at screening Pediatric patients with r/r B ALL r/r CD19-positive aggressive mature B including the B NHL subtypes: i) diffuse large B cell lymphoma, ii) Burkitt's lymphoma, iii) primary mediastinal large B cell lymphoma, iv) high-grade B cell lymphoma (not otherwise specified). * Karnofsky (age ≥ 10 years) or Lansky (age \< 10 year) performance status score ≥ 50%. * In participants with B ALL, local documentation of CD19 expression on leukemic blasts in the BM, peripheral blood, or cerebrospinal fluid or biopsy done no…
Interventions
- BiologicalAUTO1
Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells
Locations (8)
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Methodist Children's HospitalSan Antonio, Texas
- Primary Children's HospitalSalt Lake City, Utah
- Hospital Vall d'HebronBarcelona
- Hospital Nino JesusMadrid
- Great Ormond Street Hospital for Children NHS Foundation TrustLondon